CYSTIC FIBROSIS

This study tested the drug Trikafta in 42 people with cystic fibrosis who have rare gene changes not normally treated with this medicine. Participants took Trikafta for about four weeks, and researchers measured lung function and sweat chloride levels. They also took skin or bloo…

This study looked at the long-term safety of a triple combination medicine (elexacaftor/tezacaftor/ivacaftor, also known as Trikafta) in 71 people with cystic fibrosis who were 2 years old or older. Participants had already completed a previous study of the same drug. The main go…

This study tested the safety and how the body processes the drug combination elexacaftor/tezacaftor/ivacaftor in 70 toddlers with cystic fibrosis who are 12 to 24 months old. The drug is already used in older patients to help the lungs work better. Researchers measured drug level…

This study tested whether miglustat, a drug used for other conditions, can help restore the function of a faulty protein in adults with cystic fibrosis who have the F508del mutation. Sixteen participants received miglustat and a placebo in a crossover design to measure changes in…

This study followed 852 cystic fibrosis patients aged 12 and older who were taking the drug combination ivacaftor+lumacaftor (Orkambi) for one year. Researchers wanted to see how many people stopped treatment, why, and how the drug affected lung function, nutrition, and flare-ups…

This study looked at the long-term safety of a triple combination therapy (VX-445, tezacaftor, and ivacaftor) for people with cystic fibrosis who have the F508del mutation. 507 participants who completed earlier studies were enrolled. The main goal was to check for side effects a…

This study tested a single dose of a virus mixture (bacteriophages) given intravenously to adults with cystic fibrosis who have a chronic lung infection with Pseudomonas aeruginosa. The goal was to see if the treatment is safe and can reduce the amount of bacteria in the lungs. T…

This study looked at different non-invasive methods to measure liver scarring (fibrosis) in 56 children with cystic fibrosis aged 6-18. The goal was to see which tests work best to detect early liver damage, so doctors can start treatment sooner. The methods compared include bloo…

This study tested whether a noninvasive ultrasound device called FibroScan can accurately measure liver scarring (fibrosis) in children. Currently, the only reliable method is a liver biopsy, which involves inserting a needle into the liver. The study included 264 children aged 6…

This study looked at whether CFTR modulators (like Kaftrio) improve nose and sinus symptoms and quality of life in children aged 6 to 11 with cystic fibrosis. Researchers measured symptom changes using a special questionnaire before and one year after starting treatment. The goal…

This study tested a smartphone app called MuCopilot in 70 adults with cystic fibrosis in France. The app lets patients do lung function and walking tests at home without a doctor present. The goal was to see if the app's results match standard clinic tests. If successful, the app…

This study aimed to find risk factors for osteoporosis in people with cystic fibrosis. Researchers took blood samples from 25 patients and grew bone-degrading cells in the lab to understand their potential to weaken bones. They also tested how CFTR modulators affect these bone ce…

This study looked at how often women with cystic fibrosis carry HPV, a virus that can lead to cervical cancer. Researchers followed 85 adult women, both transplant and non-transplant, for up to 24 months. They found that many had HPV or abnormal cervical cells, highlighting the n…

This study looked at sputum (phlegm) from 83 adults with cystic fibrosis who were hospitalized for a sudden worsening of lung symptoms. Researchers used genetic testing to find which antibiotic-resistant bacteria were present. The goal was to see if this approach could help docto…

This study looked at how often depression and anxiety occur in teenagers with cystic fibrosis (ages 14-17) and their parents. Researchers used simple screening questionnaires during regular clinic visits and linked the results to medical records. The goal was to find out how comm…

This study looked at how bacteria develop in the nose, mouth, and lungs of infants with cystic fibrosis during their first year of life. Researchers collected swabs and mucus samples from 7 babies to understand which bacteria are present and how they move between body sites. The …

This study talked to 27 children (ages 6 and up) who have a parent with cystic fibrosis, a serious lung disease. Researchers held interviews and small group chats led by a psychologist to learn about the children's feelings, worries, and needs. The goal is to use this information…

This study looked at whether people with cystic fibrosis (CF) have protective factors against COVID-19. Over two years, 331 children and adults with CF from European centers gave blood samples to check for SARS-CoV-2 antibodies. Researchers also tracked symptoms, lung function, a…

This study looked at whether adding an inhaled sugar solution (Bronchitol) helps clear mucus from the lungs of people with cystic fibrosis who already take a powerful medication (E/T/I). Fourteen adults with moderate to severe lung disease participated. The goal was to see if the…

This study looked at whether people with cystic fibrosis (CF) have natural protection against COVID-19. Researchers tested blood samples from 75 children and adults with CF in Europe for SARS-CoV-2 antibodies over two years. They compared antibody levels after natural infection v…

This study tested whether a voice journaling app called Kintsugi is easy and acceptable for teens aged 14-18 with cystic fibrosis to use. Twenty participants used the app for three months, and researchers tracked how often they journaled and their satisfaction. The goal was to ga…

This study looked at blood neutrophils (a type of immune cell) in 47 adults with cystic fibrosis to see how chronic infection and certain treatments affect these cells. Researchers compared cells from patients with those from healthy blood donors. The goal was to learn more about…

This study followed 79 infants with cystic fibrosis under 6 months old to see how their pancreas function changed during the first year of life. Researchers measured a stool enzyme called fecal elastase-1, which indicates how well the pancreas is working. The goal was to understa…

This study looked at whether a test using cells from the nose can predict how well the drug Orkambi works for people with cystic fibrosis. Researchers collected nasal cells from 91 participants before treatment and measured how the drug improved cell function. They then compared …